Down syndrome and leukemia: An insight into the disease biology and current treatment options

IF 6.9 2区 医学 Q1 HEMATOLOGY Blood Reviews Pub Date : 2024-03-01 DOI:10.1016/j.blre.2023.101154
Sonali P. Barwe, E. Anders Kolb, Anilkumar Gopalakrishnapillai
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Abstract

Children with Down syndrome (DS) have a 10- to 20-fold greater predisposition to develop acute leukemia compared to the general population, with a skew towards myeloid leukemia (ML-DS). While ML-DS is known to be a subtype with good outcome, patients who relapse face a dismal prognosis. Acute lymphocytic leukemia in DS (DS-ALL) is considered to have poor prognosis. The relapse rate is high in DS-ALL compared to their non-DS counterparts. We have a better understanding about the mutational spectrum of DS leukemia. Studies using animal, embryonic stem cell- and induced pluripotent stem cell-based models have shed light on the mechanism by which these mutations contribute to disease initiation and progression. In this review, we list the currently available treatment strategies for DS-leukemias along with their outcome with emphasis on challenges with chemotherapy-related toxicities in children with DS. We focus on the mechanisms of initiation and progression of leukemia in children with DS and highlight the novel molecular targets with greater success in preclinical trials that have the potential to progress to the clinic.

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唐氏综合症和白血病:对疾病生物学和当前治疗方案的洞察。
与一般人群相比,患有唐氏综合症(DS)的儿童患急性白血病的易感性高10- 20倍,并倾向于髓性白血病(ML-DS)。虽然已知ML-DS是一种预后良好的亚型,但复发的患者预后不佳。急性淋巴细胞白血病(DS- all)被认为预后较差。与非ds相比,DS-ALL的复发率很高。我们对DS白血病的突变谱有了更好的了解。利用动物、胚胎干细胞和诱导多能干细胞为基础的模型进行的研究揭示了这些突变促进疾病发生和进展的机制。在这篇综述中,我们列出了DS-白血病目前可用的治疗策略及其结果,重点是DS-白血病儿童化疗相关毒性的挑战。我们专注于DS儿童白血病的发生和发展机制,并强调在临床前试验中取得更大成功的新分子靶点,这些靶点有可能进入临床。
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来源期刊
Blood Reviews
Blood Reviews 医学-血液学
CiteScore
13.80
自引率
1.40%
发文量
78
期刊介绍: Blood Reviews, a highly regarded international journal, serves as a vital information hub, offering comprehensive evaluations of clinical practices and research insights from esteemed experts. Specially commissioned, peer-reviewed articles authored by leading researchers and practitioners ensure extensive global coverage across all sub-specialties of hematology.
期刊最新文献
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